BioMarin Bolsters Enzyme Therapy Portfolio with $270 Million Acquisition of Rare Disease Specialist Inozyme Pharma
BioMarin Pharmaceutical, a leading biotech company focused on rare diseases and enzyme replacement therapies, announced on Friday that it is acquiring Inozyme Pharma, a clinical-stage biotech specialized in rare metabolic diseases, for $270 million. This strategic acquisition aims to expand BioMarin’s enzyme expertise and pipeline, particularly with Inozyme’s late-stage investigational therapy INZ-701, which targets the ultra-rare and life-threatening disease ENPP1 deficiency.
Acquisition Details and Financial Terms
Under the terms of the agreement, BioMarin will pay $4 in cash for each outstanding share of Inozyme Pharma, valuing the company at approximately $270 million. The deal is expected to close in the third quarter of 2025, subject to customary closing conditions including anti-trust regulatory approvals and the tender of a majority of Inozyme’s shares by its stockholders. The Inozyme Board has issued a unanimous recommendation that shareholders tender their shares to BioMarin, signaling strong internal support for the transaction.
This acquisition marks a significant move by BioMarin to reinforce its leadership in enzyme replacement therapies by adding a promising candidate that addresses a serious unmet medical need in the rare disease space.
The Focus: INZ-701 for ENPP1 Deficiency
At the heart of this deal is INZ-701, Inozyme’s flagship investigational therapy currently in late-stage clinical development for ENPP1 deficiency. This rare genetic disorder is caused by mutations in the ENPP1 gene, which leads to a deficiency of the ENPP1 enzyme—a critical regulator of mineralization in the body. Patients with ENPP1 deficiency suffer from progressive and severe symptoms that affect multiple organ systems and significantly diminish quality of life.
ENPP1 deficiency manifests with early-onset osteoporosis and a heightened risk of bone fractures, as well as pathological calcification of soft tissues including tendons and arteries. The disease’s impact extends to increased cardiovascular mortality risk, severe rickets in children, and softening of bones, all of which can result in debilitating complications and premature death. Currently, there are no FDA-approved treatments specifically for ENPP1 deficiency, making INZ-701 a potentially transformative therapy for affected patients.
Mechanism of Action and Therapeutic Potential
INZ-701 is an innovative enzyme replacement therapy designed to replace the deficient ENPP1 enzyme in patients. Administered via subcutaneous injection, INZ-701 combines the active enzymatic site of the human ENPP1 protein with a fragment of a human antibody. This fusion enhances the molecule’s stability and allows it to circulate systemically, effectively reaching the tissues where enzyme activity is needed.
According to preclinical studies shared by Inozyme, INZ-701 has demonstrated the ability to prevent abnormal mineralization in models of ENPP1 deficiency, supporting its mechanism of action. By restoring ENPP1 enzyme activity, INZ-701 aims to correct the underlying cause of the disease rather than merely managing symptoms, a critical distinction in rare disease therapeutics.
Beyond ENPP1 deficiency, Inozyme is also exploring INZ-701’s potential in treating related rare conditions such as ABCC6 deficiency and calcific uremic arteriolopathy—both of which involve pathological calcification processes. These indications, although rare, represent significant unmet medical needs and could expand the market opportunity for INZ-701 if clinical development proves successful.
Context: BioMarin’s Recent Challenges and Strategic Reorganization
The acquisition comes at a pivotal time for BioMarin, which has been undergoing a period of restructuring and strategic refocusing. Last year, the company revamped its executive leadership team, reduced its workforce by approximately 225 employees, and discontinued development of four drug candidates as part of a broader effort to streamline operations and focus on high-priority programs.
Alexander Hardy, BioMarin’s President and CEO, framed the Inozyme acquisition as part of a wider corporate transformation. “As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation,” Hardy said in the announcement. He emphasized BioMarin’s strong financial position as enabling it to “bring in additional assets as we accelerate the development of medicines for patients with significant unmet need.”
Financial Performance and Growth Outlook
The timing of the acquisition follows BioMarin’s solid first-quarter 2025 earnings report, which saw the company generate $745 million in revenue—a 15% increase year-over-year and ahead of analyst expectations of $738.1 million. Much of this growth was driven by BioMarin’s existing enzyme therapy portfolio, which accounted for $484 million of the quarter’s revenues, representing an 8% increase compared to the same quarter last year.
One of BioMarin’s key growth drivers remains Voxzogo (vosoritide), a C-type natriuretic peptide analog approved for the treatment of achondroplasia in children with open growth plates. Voxzogo revenue surged 40% year-over-year to $214 million in the quarter, underscoring its market momentum and importance to BioMarin’s future growth.
Following this strong start, BioMarin reaffirmed its full-year 2025 revenue outlook, forecasting total revenues of approximately $3.1 billion, with Voxzogo expected to contribute at least $900 million. This optimistic forecast underpins the company’s confidence in its core assets as well as its pipeline expansion strategy, which now includes Inozyme’s enzyme replacement therapy candidates.
Analyst Perspectives and Market Sentiment
Despite the upbeat financial performance and strategic acquisition, some industry analysts remain cautious regarding BioMarin’s longer-term revenue sustainability. A May 2 report by William Blair analysts highlighted uncertainties surrounding the company’s current revenue drivers and questioned whether these assets have the potential to deliver significant value creation in the near term.
This caution reflects broader challenges in the rare disease biotech sector, where commercial success depends heavily on regulatory approvals, market adoption, and continued pipeline innovation. The addition of INZ-701 and its potential to address a critical unmet medical need could help mitigate these concerns by diversifying BioMarin’s pipeline and reinforcing its leadership in enzyme replacement therapies.
Broader Implications for Rare Disease Treatment
The acquisition of Inozyme and its lead program INZ-701 exemplifies the growing interest among biotech companies in developing therapies for ultra-rare diseases. These conditions often lack effective treatments, and patients face limited options, creating a pressing need for innovative therapies.
BioMarin’s investment reflects a strategic commitment to rare disease innovation, leveraging its expertise in enzyme replacement technologies to deliver transformative treatments. If successful, INZ-701 could set a new standard of care for ENPP1 deficiency and related disorders, offering hope to patients and families affected by these devastating conditions.
In summary, BioMarin’s $270 million acquisition of Inozyme Pharma represents a calculated move to deepen its enzyme therapy portfolio and accelerate development of promising treatments for rare diseases. The deal highlights the strategic importance of INZ-701, a novel enzyme replacement therapy targeting the unmet needs of patients with ENPP1 deficiency—a rare, life-altering genetic disorder with no approved therapies.
As BioMarin continues its corporate transformation and focuses on both internal innovation and external partnerships, this acquisition positions the company to build on its recent financial momentum and advance its mission of delivering meaningful medicines to patients with serious unmet needs. The coming months and clinical data readouts will be crucial to validating INZ-701’s therapeutic potential and assessing how it complements BioMarin’s existing pipeline and commercial franchises.
